Meeting round-up: ISPOR Europe 2019

For many health economists, November is ISPOR Europe month, and this year was no exception! We gathered in the fantastic Bella Center in Copenhagen to debate, listen and breathe health economics and outcomes research from the 2nd to the 6th November. Missed it? Would like a recap? Stay tuned for the #ISPOREurope 2019 round-up!

Bella Center

My ISPOR week started with the fascinating course ‘Tools for reproducible real-world data analysis’ by Blythe Adamson and Rachael Sorg. My key take-home messages? Use an interface like R-markdown to produce a document with code and results automatically. Use a version control platform like Phabricator to make code review easy. Write a detailed protocol, write the code to follow the protocol, and then check the code side by side with the protocol.

Monday started with the impressive workshop on translating oncology clinical trial endpoints to real-world data (RWD) for decision making.

Keith Abrams set the scene. Electronic health records (EHRs) may be used to derive the overall survival (OS) benefit given the observed benefit on progression-free survival (PFS). Sylwia Bujkiewicz showed an example where a bivariate meta-analysis of RCTs was used to estimate the surrogate relationship between PFS and OS (paper here). Jessica Davies discussed some of the challenges, such as the lack of data on exposure to treatments in a way that matches the data recorded in trials. Federico Felizzi presented a method to determine the optimal treatment duration of a cancer drug (see here for the code).

Next up, the Women in HEOR session! Women in HEOR is an ISPOR initiative that aims to support the growth, development, and contribution of women. It included various initiatives at ISPOR Europe, such as dinners, receptions and, of course, this session.

Shelby Reed introduced, and Olivia Wu presented on the overwhelming evidence on the benefits of diversity and on how to foster it in our work environment. Nancy Berg presented on ISPOR’s commitment to diversity and equality. We then heard from Sabina Hutchison about how to network in a conference environment, how to develop a personal brand and present our pitch. Have a look at my twitter thread for the tips. For more information on the Women in HEOR activities at ISPOR Europe, search #WomenInHEOR on twitter. Loads of cool information!

My Monday afternoon started with the provocatively titled ‘Time for change? Has time come for the pharma industry to accept modest prices?’. Have a look here for my live twitter thread. Kate Dion started by noting that the pressure is on for the pharmaceutical industry to reduce drug prices. Sarah Garner argued that lower prices lead to more patients being able to access the drug, which in turn increases the company’s income. Michael Schröter argued that innovative products should have a premium price, such as with Hemlibra. Lastly, Jens Grueger supported the implementation of value-based price, given the cost-effectiveness threshold.

Keeping with the drug pricing theme, my next session was on indication-based pricing. Mireia Jofre Bonet tackled the question of whether a single price is stifling innovation. Adrian Towse was supportive of indication-based pricing because it allows for the price to depend on the value of each indication and expand access to the full licensed population. Andrew Briggs argued against indication-based pricing for three reasons. First, it would give companies the maximum value-based price across all indications. Second, it would lead to greater drug expenditure, leading to greater opportunity costs. Third, it would be difficult to enforce, given that it would require cooperation of all payers. Francis Arickx explained the pricing system in Belgium. Remarkably, prices can be renegotiated over time depending on new entrants to market and new evidence. Another excellent session at ISPOR Europe!

My final session on Monday was about the timely and important topic of approaches for OS extrapolation. Elisabeth Fenwick introduced the session by noting that innovations in oncology have given rise to different patterns of survival, with implications for extrapolation. Sven Klijn presented on the various available methods for survival extrapolation. John Whalen focused on mixture cure models for cost-effectiveness analysis. Steve Palmer argued that, although new methods, such as mixture cure models, may provide additional insight, the approach should be justified, evidence-based and alternatives explored. In sum, there is no single optimal method.

On Tuesday, my first session was the impressive workshop on estimating cost-effectiveness thresholds based on the opportunity cost (twitter thread). Nancy Devlin set the scene by explaining the importance of getting the cost-effectiveness threshold right. James Lomas explained how to estimate the opportunity cost to the health care system following the seminal work by Karl Claxton et al and also touching on some of James’s recent work. Martin Henriksson noted that, by itself, the opportunity cost is not sufficient to define the threshold if we wish to consider solidarity and need alongside cost-effectiveness. The advantage of knowing the opportunity cost is that we can make informed trade-offs between health maximisation and other elements of value. Danny Palnoch finished the panel by explaining the challenges when deciding what to pay for a new treatment.

Clearly there is a tension between the price that pharmaceutical companies feel is reasonable, the opportunity cost to the health care service, and the desire by stakeholders to use the drug. I feel this in every session of the NICE appraisal committee!

My next session was the compelling panel on the use of RWD to revisit the HTA decision (twitter thread). Craig Brooks-Rooney noted that, as regulators increasingly license technologies based on weaker evidence, HTA agencies are under pressure to adapt their methods to the available evidence. Adrian Towse proposed a conceptual framework to use RWD to revisit decisions based on value of information analysis. Jeanette Kusel went through examples where RWD has been used to inform NICE decisions, such as brentuximab vendotin. Anna Halliday discussed the many practical challenges to implement RWD collection to inform re-appraisals. Anna finished with the caution against prolonging negotiations and appraisals, which could lead to delays to patient access.

My Wednesday started with the stimulating panel on drugs with tumour agnostic indications. Clarissa Higuchi Zerbini introduced the panel and proposed some questions to be addressed. Rosa Giuliani contributed with the clinical perspective. Jacoline Bouvy discussed the challenges faced by NICE and ways forward in appraising tumour-agnostic drugs. Marc van den Bulcke finished the panel with an overview of how next generation sequencing has been implemented in Belgium.

My last session was the brilliant workshop on HTA methods for antibiotics.

Mark Sculpher introduced the topic. Antibiotic resistance is a major challenge for humanity, but the development of new antibiotics is declining. Beth Woods presented a new framework for HTA of antibiotics. The goal is to reflect the full value of antibiotics whilst accounting for the opportunity cost and uncertainties in the evidence (see this report for more details). Angela Blake offered the industry perspective. She argued that revenues should be delinked to volume, to be holistic in the value assessment, and to be mindful of the incentives faced by drug companies. Nick Crabb finished by introducing a new project, by NICE and NHS England, on the feasibility of innovative value assessments for antibiotics.

And this is the end of the absolutely outstanding ISPOR Europe 2019! If you’re eager for more, have a look at the video below with my conference highlights!

Advancing the science of value assessment: new open-source model for non-small cell lung cancer

The OSVP is designed to improve how we measure value through an iterative and transparent process driven by input from all health care stakeholders. Join us for a webinar to better understand how the OSVP process works, learn about our recently released model in non-small cell lung cancer, and find out how to get involved. Please click here to register and reserve your spot!


Chris Sampson’s journal round-up for 11th June 2018

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

End-of-life healthcare expenditure: testing economic explanations using a discrete choice experiment. Journal of Health Economics Published 7th June 2018

People incur a lot of health care costs at the end of life, despite the fact that – by definition – they aren’t going to get much value from it (so long as we’re using QALYs, anyway). In a 2007 paper, Gary Becker and colleagues put forward a theory for the high value of life and high expenditure on health care at the end of life. This article sets out to test a set of hypotheses derived from this theory, namely: i) higher willingness-to-pay (WTP) for health care with proximity to death, ii) higher WTP with greater chance of survival, iii) societal WTP exceeds individual WTP due to altruism, and iv) societal WTP may exceed individual WTP due to an aversion to restricting access to new end-of-life care. A further set of hypotheses relating to the ‘pain of risk-bearing’ is also tested. The authors conducted an online discrete choice experiment (DCE) with 1,529 Swiss residents, which asked respondents to suppose that they had terminal cancer and was designed to elicit WTP for a life-prolonging novel cancer drug. Attributes in the DCE included survival, quality of life, and ‘hope’ (chance of being cured). Individual WTP – using out-of-pocket costs – and societal WTP – based on social health insurance – were both estimated. The overall finding is that the hypotheses are on the whole true, at least in part. But the fact is that different people have different preferences – the authors note that “preferences with regard to end-of-life treatment are very heterogeneous”. The findings provide evidence to explain the prevailing high level of expenditure in end of life (cancer) care. But the questions remain of what we can or should do about it, if anything.

Valuation of preference-based measures: can existing preference data be used to generate better estimates? Health and Quality of Life Outcomes [PubMed] Published 5th June 2018

The EuroQol website lists EQ-5D-3L valuation studies for 27 countries. As the EQ-5D-5L comes into use, we’re going to see a lot of new valuation studies in the pipeline. But what if we could use data from one country’s valuation to inform another’s? The idea is that a valuation study in one country may be able to ‘borrow strength’ from another country’s valuation data. The author of this article has developed a Bayesian non-parametric model to achieve this and has previously applied it to UK and US EQ-5D valuations. But what about situations in which few data are available in the country of interest, and where the country’s cultural characteristics are substantially different. This study reports on an analysis to generate an SF-6D value set for Hong Kong, firstly using the Hong Kong values only, and secondly using the UK value set as a prior. As expected, the model which uses the UK data provided better predictions. And some of the differences in the valuation of health states are quite substantial (i.e. more than 0.1). Clearly, this could be a useful methodology, especially for small countries. But more research is needed into the implications of adopting the approach more widely.

Can a smoking ban save your heart? Health Economics [PubMed] Published 4th June 2018

Here we have another Swiss study, relating to the country’s public-place smoking bans. Exposure to tobacco smoke can have an acute and rapid impact on health to the extent that we would expect an immediate reduction in the risk of acute myocardial infarction (AMI) if a smoking ban reduces the number of people exposed. Studies have already looked at this effect, and found it to be large, but mostly with simple pre-/post- designs that don’t consider important confounding factors or prevailing trends. This study tests the hypothesis in a quasi-experimental setting, taking advantage of the fact that the 26 Swiss cantons implemented smoking bans at different times between 2007 and 2010. The authors analyse individual-level data from Swiss hospitals, estimating the impact of the smoking ban on AMI incidence, with area and time fixed effects, area-specific time trends, and unemployment. The findings show a large and robust effect of the smoking ban(s) for men, with a reduction in AMI incidence of about 11%. For women, the effect is weaker, with an average reduction of around 2%. The evidence also shows that men in low-education regions experienced the greatest benefit. What makes this an especially nice paper is that the authors bring in other data sources to help explain their findings. Panel survey data are used to demonstrate that non-smokers are likely to be the group benefitting most from smoking bans and that people working in public places and people with less education are most exposed to environmental tobacco smoke. These findings might not be generalisable to other settings. Other countries implemented more gradual policy changes and Switzerland had a particularly high baseline smoking rate. But the findings suggest that smoking bans are associated with population health benefits (and the associated cost savings) and could also help tackle health inequalities.